Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Country Product Indication Sponsor Date
Triheptanoin Long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency Ultragenyx UK Limited July 28, 2015
Allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo Duchenne muscular dystrophy Karl Rouger July 28, 2015
Triheptanoin Carnitine palmitoyltransferase II deficiency Ultragenyx UK Limited July 28, 2015
Cannabidiol Perinatal asphyxia GW Pharma Ltd July 28, 2015
Humanised IgG4 monoclonal antibody against extracellular tau Progressive supranuclear palsy Biogen Idec Limited July 28, 2015
Triheptanoin Mitochondrial trifunctional protein deficiency Ultragenyx UK Limited July 28, 2015
Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate Amyotrophic lateral sclerosis PBS Regulatory Consulting Group Limited July 28, 2015
Synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene Primary Hyperoxaluria Type 1 Dicerna EU Limited July 28, 2015
Glycyl-L-2-methylprolyl-L-glutamic acid Fragile X syndrome QRC Consultants Ltd July 28, 2015
Sarizotan hydrochloride Rett syndrome Newron Pharmaceuticals SpA July 28, 2015
Human plasminogen Plasminogen deficiency ProMetic BioTherapeutics Ltd July 28, 2015
Antroquinonol Hepatocellular Carcinoma Golden Biotechnology Corporation;10F, No. 9, Ln. 3, Tamsui Dist., New Taipei City 251; July 23, 2015
Cannabidiol West syndrome Insys Development Company, Inc.;1333 South Spectrum Boulevard, Suite 100;Chandler, Arizona, 85286 July 23, 2015
Procaspase-activating compound 1 Glioma Vanquish Oncology, Inc.;2001 S. First Street, Suite 201;Champaign, Illinois, 61820 July 23, 2015
Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex Glioma Ziopharm Oncology, Inc.;One First Avenue, Navy Yard Plaza;Boston, Massachusetts, 02129 July 23, 2015
Mongersen pediatric Crohn’s disease Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 July 20, 2015
Amantadine HCl Levodopa-induced dyskinesia Osmotica Pharmaceutical Corporation;Regulatory, Clinical Operations & Legal, Lumina Station #2, Suite 209-A;Wilmington, North Carolina, 28403 July 20, 2015
Adeno-associated virus serotype 9 vector containing human Iduronate-2-sulfatase transgene Hunter Syndrome Laboratorios del Dr. Esteve, S.A.;Av. Marie de Deu de Montserrat, 221;Barcelona July 16, 2015
Recombinant humanized anti-matrix metalloprotein 9 (MMP9) monoclonal antibody IgG4 Stomach Cancer Gilead Sciences, Inc.;199 E. Blaine St.;Seattle, Washington, 98102 July 16, 2015
Ombitasvir/paritaprevir/ritonavir and dasabuvir Pediatric patients with hepatitis C virus infection (0 through 16 years of age) Abbvie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 July 16, 2015
DPX-Survivac Ovarian cancer Immunovaccine Technologies, Inc.;1344 Summer Street, Halifax, Nova Scotia B3H; July 14, 2015
Anisina Neuroblastoma Novogen Ltd;16-20 Edgeworth David Avenue, Hornsbsy, NSW 2077; July 14, 2015
Adenovirus vaccine encoding reduced expression in immortalized cell protein Malignant pleural mesothelioma MTG Biotherapeutics;8195 Run of the Knolls Court;San Diego, California, 92127 July 14, 2015
Cannabidiol Graft Versus Host Disease Talent biotechs Ltd.;14 - 18 Baruch Hirsch St., Bnei Brak 5120206; July 14, 2015
Melphalan Bile duct cancer Delcath Systems, Inc.;1633 Broadway, Suite 22C;New York, New York, 10019 July 14, 2015